corporate overview april 2017 - enterisbiopharma.com · 35+ years of pharma, medtech and biotech...
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Corporate OverviewApril 2017
NON-CONFIDENTIAL
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Internal Pipeline - to be licensed to a partner after POC
• Lead product candidate, Ovarest™ (oral leuprolide tablet)• Initial indication: Endometriosis• Phase 2A starting Q2 2017 (POC)• Estimated at >$600M in the United States for endometriosis only
• Tobrate™ (oral tobramycin tablet) - fast track and QIDP status granted by FDA• Initial indication uUTI• Currently in formulation optimization• Estimated at >$400M in the United States for uUTI
Clinical / Commercial Tablet Manufacturing
• Experienced formulation development and clinical trial manufacturer supporting our partners from feasibility through Phase 2 clinical trial supplies, including analytical development and stability testing
• Currently developing the capability for Phase 3 and commercial manufacturing of products utilizing our formulation technology.
Peptelligence™ PlatformProprietary, Cutting-Edge Oral Drug Delivery Technology
• Novel formulation technology enables oral delivery of molecules that are typically injected, including peptides and BCS class II, III, and IV small molecules
• Robust IP protection in the U.S. and abroad through 2030
Feasibility-to-Licensing Partnering Program
• Most advanced projects using our oral drug delivery technology:• Tarsa Therapeutics – NDA submitted for TBRIA™ in osteoporosis• Cara Therapeutics – Phase 2B initiated Q3 2016 in chronic pain
(osteoarthritis) and Phase 1 uremic pruritus data expected this quarter• Ferring Pharmaceuticals – License agreement to develop an oral formulation
executed Q1 2017
Enteris BioPharmaOverview
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Leadership History• Joel Tune
Executive Chairman & Chief Executive Officer 35+ years of pharma, medtech and biotech experience Extensive management experience in product development,
sales and marketing, strategy, M&A and general management Successfully completed more than 20 business development
transactions for Baxter Healthcare’s Medication DeliveryGroup
• Brian ZietsmanPresident & Chief Financial Officer 25+ years of strategic finance and accounting experience Diverse financial capacities with public and private
pharma/biotech companies
• Paul Shields, Ph.D.Chief Operating Officer 24+ years in the biopharma industry Extensive Chemistry, Manufacturing, and Controls (CMC)
experience
Enteris BioPharmaExecutive Leadership
Overview of Peptelligence™Intelligent Solutions for Oral Drug Delivery™
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• Reduces peptide degradation
• Increases paracellular transport
• No modification of the peptide required
• Permeation enhancer acts as a surfactant
• Paracellular transport bypasses transcellular permeation hurdles
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Oral Peptide Delivery Challenges• GI tract degrades and digests
peptides• Low permeability through the
intestinal cell layer
Oral Small Molecule Delivery Challenges• Solubility or dissolution with
limited absorption• Poor permeability due to
interaction with efflux transporters or other mechanisms
Peptelligence™ Solution
Peptelligence™ The Peptelligence™ Solution
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Increases product uptake & use vs. parenteral administration
• Increases physician starts• Improves patient compliance• Expands indications
Protects & extends product exclusivity & commercial life
• Provides compelling differentiation in competitive markets
• Adds extra layer of robust IP protection through 2030
Mechanism for Oral Drug Delivery
Enteric coat prevents tablet from opening in stomach at low pH
API absorbed across intestinal wall via paracellular transport
Enteric coat dissolves at neutral pH in the small intestine
Protease inhibitor, permeability enhancers and API released
Peptellgence™Mechanism for Oral Drug Delivery
Abso
lute
Bio
avai
labi
lity
(%)
Molecular Weight (Da)7
The Enteris technology has enabledthe oral delivery of several peptidesand small molecules across a rangeof physical and chemical properties.
Bioavailability data from dogs
Peptellgence™Bioavailability Across a Range of Molecular Weights
8Enteris has conducted more than 25 pre-clinical feasibility programs
NON-CONFIDENTIAL
Peptellgence™Clinical Development Pipeline
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Robust IP Protection
9 issued U.S. patents2 allowed U.S. patent applications
3 pending U.S. provisional patent applications
Key issued patents extend through at least 2030
29 issued Foreign patents5 pending Foreign patent applications
Peptellgence™Patent Portfolio
Enteris Clinical AssetsInnovative Oral Therapeutics for Women’s Health
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Peptelligence™ Solution • Ovarest™ (oral leuprolide tablet), our leading product candidate isbeing evaluated for its potential to treat endometriosis
• Extensive market research and clinician feedback indicates thatdaily oral leuprolide tablet would offer patient friendly alternative tomonthly depot injections and drive Ovarest™ use early and moreoften
• Estimated at >$600M in the United States for endometriosis
Clinical Development • Crossover PK/food effect Phase 1 study completed in 14 healthyvolunteers
• Phase 2a parallel group PK/PD study in 38 healthy volunteers to beinitiated Q1 2017
Need • 6 million U.S. women have endometriosis• Limited efficacy with oral and injectable hormonal contraception• LUPRON DEPOT® (leuprolide acetate for depot suspension) most
efficacious treatment; No oral formulation exists on the market
Ovarest™Oral Leuprolide Tablet for the Treatment of Endometriosis
12*This plan has incorporated FDA (DBRUP) recommendations per PIND Meeting 125004 held on Dec. 15, 2014.
Phase 1Crossover PK/Food Effect study in 14 healthy volunteers
2 Ovarest doses vs 1 mg SC
Phase 2aParallel group
PK/PD study in 38 healthy volunteers
4 mg tablet QD; 4 mg tablet BID; depot for 1 month
Phase 3Parallel-group, placebo-controlled, double-blind studies in women with
endometriosis
Registration Trials (Two Phase 3 Efficacy/Safety Studies)Human POC: PK
PK/PD Modeling
PK/PD Bridging to Lupron Depot®
Ovarest™Clinical Development Plan
Complete Study Start: Q2 2017Data Read out: Q4 2017
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Peptelligence™ Solution • Tobrate™ (oral tobramycin tablet) is being evaluated for its potentialto treat uUTI
• Tobramycin is an ideal candidate for uUTI as it concentrates in theurine after dosing – enables lower dose
• Low dose / short duration improves safety profile• Estimated at >$400M in sales in the United States, even as
primarily a second line agent
Clinical Development • Fast track and QIDP status granted by FDA• Currently in formulation optimization.
Need • Uncomplicated urinary tract infections (uUTI) are highly prevalent,affecting 10 million U.S. women each year
• Currently available first-line therapies are becoming increasinglyineffective due to antibiotic resistance
• Tobramycin, which has minimal resistance issues, is available in avariety of dosage forms but no oral formulation exists on the market
Tobrate™Oral Tobramycin Tablet for the Treatment of uUTI
Partner Clinical AssetsProviding Advanced Oral Formulation Solutions to Build Long-Term, Strategic Partnerships
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Enteris BioPharmaPartners Receive
The only technology in the industry that has demonstrated broad
applicability, effectiveness, and scalability
Manufacturing expertise and infrastructure to scale production up to small commercial scale
The only technology with predictable performance: reliable estimation of likelihood of success based on API
physical-chemical properties
Peptellgence™Key Differentiators
• GMP facility• Strong inspection history (no 483s
issued in last 3 FDA inspections)• Most recent inspection: Jan. 2016
• Manufacture of Phase 1 and 2 clinical trial material
• Developing capacity for 50-250kg commercial batches
• Dedicated R&D, process development, tableting and film-coating suites
• Full QA/QC
• Analytical development, release and stability
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Peptellgence™Clinical Trial / Commercial Tablet Manufacturing Capability
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Peptelligence™ Solution • TBRIA™ employs our proprietary technology, allowing for once-daily oral delivery of calcitonin
• Provides a more convenient route of administration for patients andmay result in better patient compliance
Clinical Development • Phase 3 ORACAL trial showed TBRIA™ was superior to both nasalcalcitonin spray and placebo in increasing bone mineral densityafter 48 weeks
• NDA submitted in October 2015 for the treatment ofpostmenopausal osteoporosis in women greater than 5 yearspostmenopause when alternative treatments unsuitable
Need • Injectable and intranasal calcitonin has been used for the treatmentof osteoporosis for more than 35 years
• Osteoporosis patients either abandon or are reluctant to initiatetreatment due to medication interfering with their lives or side effectconcerns
Tarsa Therapeutics & TBRIA™Overview
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Peptelligence™ Solution • Oral CR845 tablet formulation synthesized using our proprietaryPeptelligence™ technology platform
• Oral delivery of peripherally selective kappa opioid agonist haspotential to provide continuity of care for acute and chronic painpatients
Clinical Development • Positive Phase 2a for OA trial results reported in Q4 2015• Double-blind, placebo-controlled Phase 2b for OA trial initiated
September 2016
Need • Currently marketed opioids inhibit intestinal transit and carry a riskof causing addiction and life-threatening respiratory depression
• 79% of patients report adverse events from pain medications –most adverse events are opioid-related
• Significant need among osteoarthritis (OA) and general chronic painpatients for new treatment options
Cara Therapeutics & Oral CR845™Osteoarthritis and General Chronic Pain
• Confirmed Analgesic Exposure at 1mg-5mg Tablet Strength• Well Tolerated –No SAEs on Single or Repeat Dose (6 day b.i.d.)
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Cara Therapeutics & Oral CR845™Phase 1b Study Data
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5.0 mg Dose Group • 50% reported at least a 30% reduction in pain score at the end oftreatment period
• Integrated AUC analysis of overall NRS score indicated a significantreduction compared to lower doses (Wilcoxon Rank Sum Test:p=0.02)
• Pain score reduction accompanied by a significant reduction inmean rescue medication of approximately 80% (ANOVA: p=0.02)
• 59% of patients used no rescue medication in Week 2• Dose effectiveness was further supported by significant, dose-
related increases in the proportion of patients whose OA was "verymuch improved" or "much improved” (Cochran-Mantel-Haenszeltest, p=0.02, 2-sided)
Overall • Dose-related reduction in mean joint pain score (NRS) after two-weeks, ranging from -25% (0.25 mg) to -34% (5.0 mg)
• All four tablet strengths observed to be safe and well tolerated
Cara Therapeutics & Oral CR845™Positive Top-Line Results in Phase 2a Osteoarthritis Study
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Peptelligence™ Solution • Oral CR845 tablet formulation synthesized using our proprietaryPeptelligence™ technology platform
Clinical Development • Phase 1 trial for pruritus initiated in October 2016 in hemodialysispatients
Need • Uremic pruritus (UP) is an intractable systemic itch condition thatoccurs with the greatest frequency and intensity in chronic kidneydisease
• Prevalence of UP to be approximately 40 percent of patients withend-stage renal disease (ESRD), with approximately 24 percent ofpatients reporting severe pruritus.
• Moderate-to-severe chronic pruritus has repeatedly been shown todirectly decrease quality of life, contribute to symptoms that impairquality of life (such as poor sleep quality), and is associated withdepression.
Cara Therapeutics & Oral CR845™Uremic Pruritus
Thank You!